Rachel Bailey, Ph.D.
Department Center for Alzheimer’s and Neurodegenerative Diseases | Pediatrics
Graduate Programs Neuroscience
Dr. Bailey received dual Bachelor of Science degrees in Biology/Bioinformatics and Molecular Biology from Rensselaer Polytechnic Institute. She began her Ph.D. in Neuroscience at the Mayo Clinic and completed it at the University of Florida under the mentorship of Dr. Jada Lewis. Dr. Bailey’s graduate work focused on studying modifiers of tauopathies. She demonstrated that the LRRK2 protein that is linked to Parkinson’s disease directly phosphorylates tau protein at a novel epitope, which is an important step in the neurodegenerative process.
For her postdoctoral fellowship, Dr. Bailey worked in the Gene Therapy Center at the University of North Carolina Chapel Hill in the lab of Dr. Steven Gray. She contributed to the preclinical development of an AAV-based gene therapy for the rare pediatric neurodegenerative disorder, Giant Axonal Neuropathy (GAN), which is currently being tested in a Phase I clinical trial that is ongoing at the Clinical Center at the NIH (NCT02362438). Dr. Bailey also characterized autonomic nervous system dysfunction in GAN rodent models and optimized targeting AAV vector to the peripheral nervous system in these models.
Now at UT Southwestern, the research in the Bailey lab focuses on the development of gene therapies for neurological disorders and facilitates the translation of these treatments for use in human. Pediatric disorders that she is working on include SLC13A5 epileptic encephalopathy, Multiple Sulfatase Deficiency, Charcot Marie Tooth disease type 4J and GAN. Dr. Bailey has expanded her gene therapy research efforts beyond monogenic diseases to more complex neurodegenerative disorders, including tauopathies, such as Alzheimer’s disease. Her lab utilizes AAV vector engineering for both gene replacement and gene-silencing approaches, explores alternative methods of virus delivery, performs proof-of-concept studies in cell culture models, and performs IND-enabling studies in animal models for translation of treatments to the clinic.
- Developing gene therapies for Alzheimer's and neurodegenerative diseases
- Developing gene therapies for pediatric neurological disorders
- Engineering adeno-associated viral (AAV) vectors
- Selective targeting of AAV vectors to the central and peripheral nervous systems
- Translation of gene therapies to clinical trials
- Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy.
- Bailey RM, Armao D, Nagabhushan Kalburgi S, Gray SJ Mol Ther Methods Clin Dev 2018 Jun 9 160-171
- LRRK2 phosphorylates novel tau epitopes and promotes tauopathy.
- Bailey RM, Covy JP, Melrose HL, Rousseau L, Watkinson R, Knight J, Miles S, Farrer MJ, Dickson DW, Giasson BI, Lewis J Acta Neuropathol. 2013 Dec 126 6 809-27
- Advancing the pathologic phenotype of giant axonal neuropathy: early involvement of the ocular lens.
- Armao D, Bouldin TW, Bailey RM, Hooper JE, Bharucha DX, Gray SJ Orphanet J Rare Dis 2019 02 14 1 27
- Lrrk promotes tau neurotoxicity through dysregulation of actin and mitochondrial dynamics.
- Bardai FH, Ordonez DG, Bailey RM, Hamm M, Lewis J, Feany MB PLoS Biol. 2018 Dec 16 12 e2006265
- Autonomic nervous system involvement in the giant axonal neuropathy (GAN) KO mouse: implications for human disease.
- Armao D, Bailey RM, Bouldin TW, Kim Y, Gray SJ Clin. Auton. Res. 2016 08 26 4 307-13
- Physiologically relevant factors influence tau phosphorylation by leucine-rich repeat kinase 2.
- Hamm M, Bailey R, Shaw G, Yen SH, Lewis J, Giasson BI J. Neurosci. Res. 2015 Oct 93 10 1567-80
- Effects of the C57BL/6 strain background on tauopathy progression in the rTg4510 mouse model.
- Bailey RM, Howard J, Knight J, Sahara N, Dickson DW, Lewis J Mol Neurodegener 2014 Jan 9 8
- In vivo functional brain mapping in a conditional mouse model of human tauopathy (tauP301L) reveals reduced neural activity in memory formation structures.
- Perez PD, Hall G, Kimura T, Ren Y, Bailey RM, Lewis J, Febo M, Sahara N Mol Neurodegener 2013 Feb 8 9
- Neuronal sensitivity to TDP-43 overexpression is dependent on timing of induction.
- Cannon A, Yang B, Knight J, Farnham IM, Zhang Y, Wuertzer CA, D'Alton S, Lin WL, Castanedes-Casey M, Rousseau L, Scott B, Jurasic M, Howard J, Yu X, Bailey R, Sarkisian MR, Dickson DW, Petrucelli L, Lewis J Acta Neuropathol. 2012 Jun 123 6 807-23
- CHIP regulates leucine-rich repeat kinase-2 ubiquitination, degradation, and toxicity.
- Ko HS, Bailey R, Smith WW, Liu Z, Shin JH, Lee YI, Zhang YJ, Jiang H, Ross CA, Moore DJ, Patterson C, Petrucelli L, Dawson TM, Dawson VL Proc. Natl. Acad. Sci. U.S.A. 2009 Feb 106 8 2897-902
Methods for Gene Transfer to the Central Nervous System. In Advances in Genetics
Kantor B, Bailey RM, Wimberly K, Nagabhushan Kalburgi S, and Gray SJ. (2014). Cambridge, Massachusetts, Academic Press
Honors & Awards
- Finalist in Swim with the Sharks!
A Team Science Pilot Grant Competition, UT Southwestern (2018)
- NIH NRSA Postdoctoral Fellowship
- American Society for Gene and Cell Therapy
Conference Travel Scholarship (2015)
- NIH T32 Postdoctoral Fellowship
- Peripheral Nerve Society Award
Travel Scholarship (2015)
- Bryan Robinson Endowment
- NIH NRSA Pre-doctoral Fellowship
- Parkinson's Disease Summer Student Fellowship
- Robert D. and Patricia E. Kern Pre-doctoral Fellowship
- American Society for Gene and Cell Therapy (2014)
- Society for Neuroscience (2012)
- International Society to Advance Alzheimer's Research and Treatment
- Peripheral Nerve Society